Clin Pediatr Hematol Oncol.
2026 Apr;33(1):39-44. 10.15264/cpho.2026.33.1.39.
Eltrombopag-Induced Near-Fatal Hyperammonemic Encephalopathy – Case Report
- Affiliations
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- 1Paediatric General Medicine, Alder Hey Children’s NHS Foundation Trust, Liverpool, UK
- 2Paediatric Critical Care, Alder Hey Children’s NHS Foundation Trust, Liverpool, UK
- 3Laboratorio Malattie Metaboliche e Biologia del Farmaco, Ospedale Pediatrico Bambino Gesù, Roma, Italy
- 4Paediatric Radiology, Alder Hey Children’s NHS Foundation Trust, Liverpool, UK
- 5Liverpool Centre for Genomic Medicine, Liverpool Women’s NHS Foundation Trust, Liverpool, UK
- 6Manchester Centre for Genomic Medicine, St Mary's Hospital, Manchester University NHS Foundation Trust, Health Innovation Manchester, Manchester, UK
- 7Division of Evolution & Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, UK
- KMID: 2580980
- DOI: http://doi.org/10.15264/cpho.2026.33.1.39
Abstract
- An 8-year-old girl with chronic immune thrombocytopenic purpura treated with eltrombopag presented with lactic acidosis and severe hyperammonemic encephalopathy necessitating hemofiltration. Genetic metabolic disorders associated with hyperammonemia were excluded. Despite standard dosing, supratherapeutic plasma levels of eltrombopag were found, suggesting an adverse drug effect due to drug accumulation. Awareness of hyperammonemia as adverse reaction to eltrombopag is warranted. Close monitoring of transaminases is required in children and therapeutic drug monitoring could aid in tailoring effective treatment doses for children to mitigate risk in populations that are more susceptible to complications.
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